Can Suramin Emerge as a Treatment for Autism Spectrum Disorders (ASD)?

Suramin has been
used for over a century for the treatment of African sleeping sickness

A study found
that suramin may be effective in children with autism

Larger studies
are needed to prove the efficacy and safety of the drug before it can be
safely used in children with autism

The
drug suramin, used for the treatment of African sleeping sickness, could soon
find its way into the treatment armamentarium of autism spectrum disorders
(ASD) in children. A study evaluating suramin for this new
possibility was published in the Annals
of Clinical and Translational Neurology.

Medicine has evolved in
a big way over the decades. New molecules have evolved into drugs, while some
of the older, toxic and relatively inefficient drugs have fallen into disuse.
An almost reverse trend also exists, where traditional and older medications
are explored for use once again, either for the same, or
for a different condition. One such drug
is suramin,which has been used for the treatment of African
sleeping sickness for around a century, and is now being evaluated for its
possible benefits in autism patients.

Can Suramin Emerge as a Treatment for Autism Spectrum Disorders (ASD)?

Sleeping sickness or African
trypanosomiasis is a parasitic infection spread by the tsetse flies. It causes
neurological symptoms like behavioral problems, confusion and alterations in
sleep cycles. With extreme measures to control the spread of flies, the disease
burden has been reduced to a large extent, with health authorities setting
goals to eliminate the infection by 2020. Suramin is one of the drugs used to
treat sleeping sickness. It is associated with a number of side effects
including nausea, diarrhea, low blood pressure and kidney problems.

‘A drug used for around a century for sleeping sickness may provide answers to the search for treatment for autism spectrum disorders (ASD).’

A
small trial, the Suramin Autism Treatment-1 (SAT-1) trial, on 10 boys aged
between 5 and 14 years evaluated the use of low-dose suramin for autism
(ASD). ASD
include a group of disorders in children characterized by problems in
communication, language and socialization, and repetitive behaviors. The
condition arises due to genetic and environmental factors that affect the
development of the child. The researchers found clues of the possible benefits of
suramin for ASD based on studies on mice.

Five out of the ten boys
included in the study received an intravenous infusion of low-dose suramin,
while the other five received only saline, which acted as a placebo. Based on
observations and standardized interviews, the researchers found that:

The boys who
received suramin improved with respect to speech and language, social
behavior and communication, focus, calmness, repetitive behaviors and
coping skills at the end of 6 weeks following treatment. This improvement
was not observed in those administered the placebo. The positive changes
caused by suramin were obvious and noted by the parents as well.

The children who
received suramin also experienced enhanced benefits from their other
treatments like speech therapy, occupational therapy and applied
behavioral analysis

The benefits of
suramin faded after several weeks. This indicates that the effects of the
single dose are temporary and multiple doses may be required

The side effects
of suramin were minimal in the study and included the development of rash.
Again, it must be remembered that only a single low dose of suramin was
used in the study

How can a drug used for
the treatment of a parasitic infection be effective in a developmental brain
disorder? Researchers
believe that they have an answer to this as well. They believe that the cell danger response plays a role in the
development of autism, along with genetic factors and exposure to environmental
toxins. The cell danger response (CDR) is a protective mechanism by cells in
response to stress or injury. It prevents interaction of the cell with other
cells so that it does not get damaged. At times, however, the response
continues even after the stressful condition or injury resolves. This can do more
harm than good, which makes it necessary to stop it with drugs like suramin.
The oxidative stress caused by the CDR may also impact nerve cells and
circuits, thereby contributing to ASD. Suramin
appears to block the effect of adenosine triphosphate (ATP), which is released
from cells and takes part in the CDR. It thereby,
pauses the CDR and protects the nerve cells from its damaging effects.

It
would now be necessary to demonstrate whether suramin can produce its benefits
in a large number of ASD children. The dose required to maintain a
sustained response with minimal adverse effects will also need to be estimated.
If suramin does not stand the test, a similar molecule with a beneficial
effect, at the same time, with minimal side effects, could see the light of the
day.

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